PCD Research and NATA co-fund grant to advance therapy for primary ciliary dyskinesia
PCD Research and NATA announced a joint two-year research grant awarded to Professor Stephen Hart at University College London (UCL), aimed at developing mRNA therapy for primary ciliary dyskinesia.
- PCD Research and NATA have jointly awarded a £250k two-year research grant to Professor Stephen Hart at UCL to develop mRNA therapy for Primary Ciliary Dyskinesia (PCD), a rare genetic condition affecting cilia function.
- PCD affects approximately 1 in 7,500 people. This grant targets the third most common disease-causing gene CCDC39, known for causing severe clinical outcomes.
- The project aims to restore the movement of cilia (tiny hair-like structures in the lungs) by delivering mRNA to replace the faulty CCDC39 protein. If successful, this method could be a significant advance in creating more effective, targeted treatments for PCD.
HARWELL, OXFORD, UK — 29 OCTOBER 2024 — PCD Research, a UK-based charity, and the MRC nucleic acid therapy research unit, NATA, have announced a joint £250k two-year research grant awarded to Professor Stephen Hart at University College London (UCL), aimed at developing mRNA therapy for PCD. The grant will fund pioneering research focused on restoring the function of cilia—the hair-like structures that are found throughout the body—by targeting one of the most severe and common genes responsible for PCD.
PCD is a rare inherited condition that affects approximately one in 7,500 people. At present, there are no approved treatments for PCD. PCD is caused by faulty proteins required for normal cilia movement, with 56 known genes identified so far. Cilia are tiny, hair-like structures that line the airways in the lungs, nose, and other parts of the body. Their coordinated movement clears mucus, bacteria, and other particles out of the respiratory tract, preventing infections. In people with PCD, where cilia movement is impaired, mucus builds up, leading to chronic respiratory infections and permanent lung damage. Out of the known 56 genes, mutations of the CCDC39 gene are the third most common cause of PCD, but are responsible for some of the most severe clinical outcomes.
The primary objective of this grant is to develop and optimise the delivery of mRNA encoding the template for the CCDC39 protein, to functionally restore cilia movement in models lacking this vital protein.
“This collaboration marks a significant moment for PCD research. By co-funding this grant with NATA, we are taking a significant step towards addressing one of the most severe genetic mutations responsible for PCD.” said Dr Harriet Holme, Chair and Founder of PCD Research. “NATA’s extensive network of clinical and academic collaborators, and expertise in nucleic acid therapeutics, will significantly contribute to the success of this project.”
“This project represents the kind of collaborative research that NATA is proud to support. At NATA, we are focused on advancing nucleic acid therapies through inter-disciplinary and translational research.” commented Professor Nick Lench, Executive Director, NATA
Click here for more information on PCD Research.